Solid Tumors Harboring NTRK FusionPhase 1/2SCOUTNCT02637687
A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children
A Phase 1/2 study testing Larotrectinib (Vitrakvi, BAY2757556) in people with Solid Tumors Harboring NTRK Fusion, run by Bayer at 45 sites in 21 countries.
Trial summary
The study is being done to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat cancer. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Solid Tumors Harboring NTRK Fusion
- What is being tested?
- Larotrectinib (Vitrakvi, BAY2757556)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 154 participants
- Where
- 45 sitesin 21 countries
- Study dates
- 16 Dec 2015to 30 Sep 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Phase 1 (Closed):
- Dose escalation: Birth through 21 years of age at C1D1 with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies and for which no standard or available systemic curative therapy exists; OR Infants from birth and older with a diagnosis of malignancy and with a documented NTRK fusion that has progressed or was nonresponsive to available therapies, and for which no standard or available curative therapy exists; OR Patients with locally advanced infantile fibrosarcoma who would require, in the opinion of the investigator, disfiguring surgery or limb amputation to achieve a complete surgical resection. Phase I dose escalation cohorts are closed to enrollment.
- Dose expansion: In addition to the above stated inclusion criteria, patients must have a malignancy with a documented NTRK gene fusion with the exception of patients with infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer. Patients with infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer may enroll into this cohort with documentation of an ETV6 rearrangement by FISH or RT-PCR or a documented NTRK fusion by next generation sequencing.
- Phase 2:
- -- Infants from birth and older at C1D1 with a locally advanced or metastatic infantile fibrosarcoma, patients with locally advanced infantile fibrosarcoma who would require, in the opinion of the investigator, disfiguring surgery or limb amputation to achieve a complete surgical resection; OR Birth through 21 years of age at C1D1 with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies and for which no standard or available systemic curative therapy exists with a documented NTRK gene fusion (or in the case of infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer with documented ETV6 rearrangement (or NTRK3 rearrangement after discussion with the sponsor) by FISH or RT-PCR. Patients with NTRK-fusion positive benign tumors are also eligible; OR Potential patients older than 21 years of age with a tumor diagnosis with histology typical of a pediatric patient and an NTRK fusion may be considered for enrollment following discussion between the local site Investigator and the Sponsor.
- Patients with primary CNS tumors or cerebral metastasis
Show all 9Show fewer
- Karnofsky (those 16 years and older) or Lansky (those younger than 16 years) performance score of at least 50.
- Adequate hematologic function
- Adequate hepatic and renal function
Who cannot join
- Major surgery within 14 days (2 weeks) prior to C1D1
- Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to C1D1, ongoing cardiomyopathy; current prolonged QTc interval > 480 milliseconds
- Active uncontrolled systemic bacterial, viral, or fungal infection
- Current treatment with a strong CYP3A4 inhibitor or inducer. Enzyme-inducing anti-epileptic drugs (EIAEDs) and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed.
- Phase 2 only:
- Prior progression while receiving approved or investigational tyrosine kinase inhibitors targeting TRK, including entrectinib, crizotinib and lestaurtinib. Patients who received a TRK inhibitor for less than 28 days of treatment and discontinued because of intolerance remain eligible.
Trial locations and contacts
45 sites in 21 countries.
45 sites in 21 countries
United States11 sites
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Cincinnati Children's Hospital Medical Center | Division of Nephrology and Hypertension
Cincinnati, Ohio, 45229
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Children's Hospital Los Angeles - Hematology/Oncology
Los Angeles, California, 90027
UCLA Jonsson Comprehensive Cancer Center
Los Angeles, California, 90095-1781
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Memorial Sloan Kettering Cancer Center New York - Main Campus
New York, New York, 10065
Nemours Children's Hospital - Florida - Hematology / Oncology
Orlando, Florida, 32827
Lucille Packard Children's Hospital Stanford - Pediatric Nephrology
Palo Alto, California, 94304
Children's Hospital of Philadelphia - Hematology/Oncology
Philadelphia, Pennsylvania, 19104
Seattle Children's Hosptial - Oncology
Seattle, Washington, 98105
Australia3 sites
Women's and Children's Hospital
North Adelaide, South Australia, 5006
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Sydney Children's Hospital
Sydney, New South Wales, 2031
Canada3 sites
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
The Hospital for Sick Children (SickKids)
Toronto, Ontario, M5G 1X8
BC Children's Hospital - Hematology/Oncology
Vancouver, British Columbia, V6H 3N1
China3 sites
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100045
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, 510000
Tianjin Medical University Cancer Institute & Hospital
Tianjin, 300000
Germany3 sites
Charité - Campus Virchow-Klinikum (CVK), Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie
Berlin, 13353
Universitaetsklinikum Heidelberg - KiTZ | Klinik für Paediatrische Onkologie, Haematologie, Immunologie und Pneumologie
Heidelberg, Baden-Wurttemberg, 69120
KLINIKUM STUTTGART - Olgahospital | Paediatrie 5 (Onkologie, Haematologie, Immunologie)
Stuttgart, Baden-Wurttemberg, 70174
Japan3 sites
National Cancer Center Hospital
Chuo-ku, Tokyo, 104-0045
Kyushu University Hospital
Fukuoka, 812-8582
Kanagawa Children's Medical Center
Yokohama, Kanagawa, 252-8555
Czechia2 sites
FN Brno - Detska nemocnice
Brno, 613 00
Fakultni nemocnice v Motole
Prague, 150 06
France2 sites
Institut Curie - Ulm - Paris
Paris, 75248
Gustave Roussy - Departement Oncologie-Radiotherapie
Villejuif, 94805
South Korea2 sites
Severance Hospital, Yonsei University Health System
Seoul, Seoul Teugbyeolsi, 03722
Seoul National University Hospital
Seoul, Seoul Teugbyeolsi, 3080
Ukraine2 sites
Governmental Noncommercial Institution "National Cancer Institute
Kyiv, 03022
Western Ukrainian Specialized Pediatric Medial Centre, Surgical Department
Lviv, 79035
Israel1 site
Clalit Health Services Schneider Children's Medical Center
Petah Tikva, 4920235
Italy1 site
Fondazione IRCCS Istituto Nazionale dei Tumori - S. C. Pediatria Oncologica
Milan, Lombardy, 20133
Spain1 site
Ciutat Sanitaria i Universitaria de la Vall d'Hebron
Barcelona, 08035
Turkey (Türkiye)1 site
Istanbul Universitesi Istanbul Tip Fakultesi
Istanbul, 34093
United Kingdom1 site
Royal Marsden NHS Trust (Surrey)
Sutton, Surrey, SM2 5PT
About this study
Official title: A Phase 1/2 Study of the Oral TRK Inhibitor Larotrectinib in Pediatric Patients With Advanced Solid or Primary Central Nervous System Tumors
The study is being done to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat cancer.
The first study part (Phase 1) is done to determine what dose level of larotrectinib is safe for children, how the drug is absorbed and changed by their bodies and how well the cancer responds to the drug. The main purpose of the second study part (Phase 2) is to investigate how well and how long different cancer types respond to the treatment with larotrectininb.
- Conditions
- Solid Tumors Harboring NTRK Fusion
- Being tested
- Larotrectinib (Vitrakvi, BAY2757556)
- Keywords
- Advanced solid tumors, Central nervous system (CNS) tumor, Extra-cranial tumor, Infantile fibrosarcoma (IFS), Neurotrophic tyrosine receptor kinase (NTRK), NTRK1, NTRK2, NTRK3, Fusion Positive, TRK fusion, TRKA, TRKB, TRKC, ETV6
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Larotrectinib (Vitrakvi, BAY2757556)Drug
Also called LOXO-101
BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1 dose escalation | Experimental | Patients will receive the different levels of dose on Day 1 (BID in accordance with the cohort assignment). Each cycle will consist of 28 days of continuous dosing.
Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. (arm closed) Receives: Drug: Larotrectinib (Vitrakvi, BAY2757556) |
| Phase 1 dose expansion | Experimental | Patients who are enrolled in the expansion cohort, following the formal dose escalation phase of the study.
Distinct from the Phase 1 dose escalation cohort, the Phase 1 expansion cohort will enroll pediatric patients with advanced solid or primary CNS tumors with a documented NTRK gene fusion, or in the case of IFS, CMN or SBC with documented ETV6 rearrangement by FISH or RT-PCR or a documented NTRK fusion by NGS.
This expansion cohort will follow the same schedule of assessments as the dose escalation cohorts. (arm closed) Receives: Drug: Larotrectinib (Vitrakvi, BAY2757556) |
| Phase 2: Patients with tumors bearing NTRK fusions (IFS)_Cohort 1 | Experimental | Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing.
Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. (arm closed) Receives: Drug: Larotrectinib (Vitrakvi, BAY2757556) |
| Phase 2: Other extra-cranial solid tumors_Cohort 2 | Experimental | Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing.
Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. (arm closed) Receives: Drug: Larotrectinib (Vitrakvi, BAY2757556) |
| Phase 2: Primary CNS tumors_Cohort 3 | Experimental | Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing.
Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. Receives: Drug: Larotrectinib (Vitrakvi, BAY2757556) |
| Phase 2: Bone health assessment_sub-cohort | Experimental | Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing.
Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation.
Patients in this group will undergo bone health assessments in addition to all other efficacy and safety assessments. Receives: Drug: Larotrectinib (Vitrakvi, BAY2757556) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Number of participants in an assigned dose cohort with treatment emergent adverse events (TEAEs) by grade assessed by NCI-CTCAE v 4.03 who experience a DLT
DLT: Dose-limiting toxicity. NCI-CTCAE: National Cancer Institute-Common Terminology Criteria for Adverse Events.
Measured: From Day 1 to Day 28 of Cycle 1 (1 Cycle=28 days)
Phase 1: Number of participants with TEAEs
Measured: From first dose of larotrectinib up to 93 months
Phase 1: Severity of TEAEs
Measured: From first dose of larotrectinib up to 93 months
Phase 2: Overall response rate (ORR) by IRRC
Proportion of participants with a best overall response of complete response (CR) or partial response (PR) as determined by an independent radiology review committee (IRRC) based on Response Evaluation Criteria in Solid Tumours (RECIST) 1.1, Response Assessment in Neuro Oncology (RANO) or International Neuroblastoma Response Criteria (INRC) as appropriate to tumor type who express NTRK gene fusions.
Measured: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death, up to 76 months
Secondary outcomes (22)
Phase 1: Maximum concentration of larotrectinib in plasma (Cmax)
Measured: Cohort 1 and 2: Cycle 1 Day 1 (C1D1) at 1 and 4 hours post-dose and C2D1 at pre-dose, and at 1 and 4 hours post-dose; Cohort 3 and Dose Expansion Cohort: C1D1 at 1 and 4 hours post-dose and C4D1 at pre-dose, 1 and 4 hours post-dose
Phase 1: Area under the concentration versus time curve from time 0 to t (AUC0-t) of larotrectinib in plasma
Measured: Cohort 1 and 2: C1D1 at 1 and 4 hours post-dose and C2D1 at pre-dose, and at 1 and 4 hours post-dose; Cohort 3 and Dose Expansion Cohort: C1D1 at 1 and 4 hours post-dose and C4D1 at pre-dose, 1 and 4 hours post-dose
Phase 1: Oral clearance (CL/F)
Measured: Cohort 1 and 2: C1D1 at 1 and 4 hours post-dose and C2D1 at pre-dose, and at 1 and 4 hours post-dose; Cohort 3 and Dose Expansion Cohort: C1D1 at 1 and 4 hours post-dose and C4D1 at pre-dose, 1 and 4 hours post-dos
Phase 1: Cerebral spinal fluid/plasma ratio of larotrectinib
Measured: C1D1 in conjunction with the post-dose 1-hour PK sample
Phase 1: Maximum tolerated dose (MTD)
Measured: From C1D1 to C1D28 of treatment of each participant in each of the assigned dose cohort, up to 16 months
Phase 1: Recommended dose for Phase 2
Measured: From the date a participants from assigned Cohort was administered the first dose to the date of the last dose for the last patient from the dose escalation phase, up to 16 months
Phase 1: Overall Response Rate (ORR)
Proportion of participants with best overall response (BOR) of CR and PR; PFS, CBR and maximum change in tumor burden as assessed based on RECIST 1.1, INRC or RANO as appropriate for tumor type by IRRC.
Measured: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 93 months
Phase 1: Mean change from baseline in Pain scores as assessed by the Wong-Baker Faces scale
Wong-Baker Faces Scale giving a pain scale between 0 (no hurt) to 10 (hurts worst).
Measured: Baseline and D1 of every cycle (1 Cycle=28 days), up to 93 months
Phase 1: Mean change in Health-related quality of life scores by PedsQL-Core
The health-related quality of life (HRQoL) is assessed with the Pediatrics Quality of Life - Core Module (PedsQL-Core) questionnaire that consists of various age-related items regarding physical, emotional, social and school functioning and gives an overall score between 0 (highest HRQoL) and 144 (lowest HRQoL).
Measured: Baseline and D1 of every cycle (1 Cycle=28 days), Up to 93 months
Phase 2: Best overall response (BOR)
Participants with best overall response (BOR) of either CR or PR determined by Investigator's or IRC's response assessment based on RANO, INRC and RECIST 1.1 as appropriate for tumor type
Measured: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 76 months
Phase 2: Duration of response (DOR)
DOR determined by 1) an independent radiology review committee and 2) the treating Investigator.
Measured: From start of first objective response of confirmed CR or PR to progression or death (due to any cause), up to 76 months
Phase 2: Proportion of patients with any tumor regression (i.e., any decrease from baseline of the longest diameters of target lesions) as a best response
Measured: From first dose of Larotrectinib, up to 76 months
Phase 2: Progression-free survival (PFS)
Measured: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 112 months
Phase 2: Overall survival (OS)
Measured: From first dose of Larotrectinib to death (due to any cause), up to 112 months
Phase 2: Number of participants with Treatment emergent adverse events (TEAEs)
Measured: From first dose of larotrectinib to discontinuation of treatment or death (due to any cayse), up to 112 months
Phase 2: Severity of adverse events as assessed by NCI-CTCAE grading V 4.03
Measured: From first dose of larotrectinib to discontinuation of treatment or death (due to any cause), up to 112 months
Phase 2: Clinical Benefit Rate (CBR)
CBR (i.e., best overall response of CR, PR or SD lasting 16 weeks or more as determined by 1) an independent radiology review committee (IRC) and 2) by the treating Investigator.
Measured: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 76 months
Phase 2: Concordance coefficient
Concordance coefficient of intra-patient molecular profile
Measured: From baseline/screening and if feasible end of treatment (EOT) and or PD and or at re-start of study treatment following a "drug holiday" and disease recurrence, up to 112 months
Phase 2: Post-operative tumor staging
Post-operative stage in patients treated with larotrectinib according to the TNM Classification of malignant tumors of the Union for International Cancer Control (UICC).
Measured: From first dose of Larotrectinib to surgical intervention, up to 112 months
Phase 2: Post-operative surgical margin assessment
Surgical margin status in patients treated with larotrectinib using the International Cancer Control (UICC)-R classification and the Intergroup Rhabdomyosarcoma Staging (IRS) systems.
Measured: From first dose of Larotrectinib to surgical intervention, up to 112 months
Phase 2: Pre-treatment surgical plan to preserve function and cosmetic outcome
Descriptive analysis of pretreatment surgical plan.
Measured: From first dose of Larotrectinib to surgical intervention, up to 112 months
Phase 2: Post-treatment plans to conserve function and cosmetic outcome
Descriptive analysis of post-treatment plans
Measured: From surgical intervention to subsequent therapy, up to 112 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2015 (estimated)
- Study start16 Dec 2015
- Primary completion20 Jul 2024
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (18)Papers the sponsor has linked to this study.
- Other trials for Solid Tumors Harboring NTRK FusionStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 154 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02637687
- Sponsor's protocol ID
- 20290
- Other IDs
- LOXO-TRK-15003, 2022-502668-20-00, 2016-003498-16, 2016
- Acronym
- SCOUT
- Status last verified
- Jun 2025
- First submitted
- 10 Dec 2015
- Last updated
- 22 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
Publications
Mascarenhas L, Ollis C, Singh Y, Menachery L, Burcoveanu DI, De La Cuesta E, van Tilburg CM, Laetsch TW. Cancer control after pausing treatment with larotrectinib in responding children with sarcoma and a TRK fusion: a plain language summary. Future Oncol. 2026 Aug 19:1-10. doi: 10.1080/14796694.2026.2711519. Online ahead of print.
PubMed 42614029Ajani JA, D'Amico TA, Bentrem DJ, Corvera CU, Das P, Enzinger PC, Enzler T, Gerdes H, Gibson MK, Grierson P, Gupta G, Hofstetter WL, Ilson DH, Jalal S, Kim S, Kleinberg LR, Klempner S, Lacy J, Lee B, Licciardi F, Lloyd S, Ly QP, Matsukuma K, McNamara M, Merkow RP, Miller AM, Mukherjee S, Mulcahy MF, Perry KA, Pimiento JM, Reddi DM, Reznik S, Roses RE, Strong VE, Su S, Uboha N, Wainberg ZA, Willett CG, Woo Y, Yoon HH, McMillian NR, Stein M. Gastric Cancer, Version 2.2025, NCCN Clinical Practice Guidelines In Oncology. J Natl Compr Canc Netw. 2025 May;23(5):169-191. doi: 10.6004/jnccn.2025.0022.
PubMed 40341199Brose MS, Westphalen CB, Pan X, Bernard-Gauthier V, Kurtinecz M, Guo H, Aris V, Brett NR, Majdi A, Subbiah V, Pennell NA, Kehl KL, Drilon A. Larotrectinib Compared With Real-World Non-Tropomyosin Receptor Kinase Inhibitor Therapies in Patients With Tropomyosin Receptor Kinase Fusion Cancer. JCO Precis Oncol. 2025 Apr;9:e2400500. doi: 10.1200/PO-24-00500. Epub 2025 Apr 23.
PubMed 40267388Mascarenhas L, DuBois SG, Albert CM, Bielack S, Orbach D, Federman N, Geoerger B, Nagasubramanian R, Zhang Y, Chisholm J, Gallego Melcon S, Goto H, Morgenstern DA, Owens C, Pappo AS, Perreault S, Schulte JH, Shukla N, Zwaan CM, Neu N, Bernard-Gauthier V, De La Cuesta E, van Tilburg CM, Laetsch TW. Elective Discontinuation of Larotrectinib in Pediatric Patients With TRK Fusion Sarcomas and Related Mesenchymal Tumors. J Clin Oncol. 2025 Apr;43(10):1180-1187. doi: 10.1200/JCO.24.00848. Epub 2025 Jan 27.
PubMed 39869835Nahon-Esteve S, Orbach D, Le Loarer F, Hofman P, Lassalle S. Fibroblastic orbital tumour with NTRK1 fusion transcript: when TRK inhibitors rescue surgery. Can J Ophthalmol. 2024 Aug;59(4):e407-e409. doi: 10.1016/j.jcjo.2023.11.008. Epub 2023 Dec 11. No abstract available.
PubMed 38096907Subbiah V, Burris HA 3rd, Kurzrock R. Revolutionizing cancer drug development: Harnessing the potential of basket trials. Cancer. 2024 Jan;130(2):186-200. doi: 10.1002/cncr.35085. Epub 2023 Nov 7.
PubMed 37934000Kummar S, Shen L, Hong DS, McDermott R, Keedy VL, Casanova M, Demetri GD, Dowlati A, Melcon SG, Lassen UN, Leyvraz S, Liu T, Moreno V, Patel J, Patil T, Mallick AB, Sousa N, Tahara M, Ziegler DS, Norenberg R, Arvis P, Brega N, Drilon A, Tan DSW. Larotrectinib efficacy and safety in adult patients with tropomyosin receptor kinase fusion sarcomas. Cancer. 2023 Dec 1;129(23):3772-3782. doi: 10.1002/cncr.35036. Epub 2023 Sep 28.
PubMed 37769113Bokemeyer C, Paracha N, Lassen U, Italiano A, Sullivan SD, Marian M, Brega N, Garcia-Foncillas J. Survival Outcomes of Patients With Tropomyosin Receptor Kinase Fusion-Positive Cancer Receiving Larotrectinib Versus Standard of Care: A Matching-Adjusted Indirect Comparison Using Real-World Data. JCO Precis Oncol. 2023 Jan;7:e2200436. doi: 10.1200/PO.22.00436.
PubMed 36689698Rudzinski ER, Drilon A, Moore A, Spinosa S, Willi M, Laetsch TW. Testing methods to diagnose TRK fusion cancer - a plain language summary and patient perspective. Future Oncol. 2022 Dec;18(38):4141-4151. doi: 10.2217/fon-2022-0863. Epub 2023 Jan 6.
PubMed 36606522Doz F, van Tilburg CM, Geoerger B, Hojgaard M, Ora I, Boni V, Capra M, Chisholm J, Chung HC, DuBois SG, Gallego-Melcon S, Gerber NU, Goto H, Grilley-Olson JE, Hansford JR, Hong DS, Italiano A, Kang HJ, Nysom K, Thorwarth A, Stefanowicz J, Tahara M, Ziegler DS, Gavrilovic IT, Norenberg R, Dima L, De La Cuesta E, Laetsch TW, Drilon A, Perreault S. Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors. Neuro Oncol. 2022 Jun 1;24(6):997-1007. doi: 10.1093/neuonc/noab274.
PubMed 34850167Bebb DG, Banerji S, Blais N, Desmeules P, Gill S, Grin A, Feilotter H, Hansen AR, Hyrcza M, Krzyzanowska M, Melosky B, Noujaim J, Purgina B, Ruether D, Simmons CE, Soulieres D, Torlakovic EE, Tsao MS. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Adults. Curr Oncol. 2021 Jan 15;28(1):523-548. doi: 10.3390/curroncol28010053.
PubMed 33467570Perreault S, Chami R, Deyell RJ, El Demellawy D, Ellezam B, Jabado N, Morgenstern DA, Narendran A, Sorensen PHB, Wasserman JD, Yip S. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Pediatric Patients. Curr Oncol. 2021 Jan 9;28(1):346-366. doi: 10.3390/curroncol28010038.
PubMed 33435412Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Muller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov;30 Suppl 8:viii31-viii35. doi: 10.1093/annonc/mdz382. Epub 2019 Dec 24.
PubMed 32223937Hong DS, DuBois SG, Kummar S, Farago AF, Albert CM, Rohrberg KS, van Tilburg CM, Nagasubramanian R, Berlin JD, Federman N, Mascarenhas L, Geoerger B, Dowlati A, Pappo AS, Bielack S, Doz F, McDermott R, Patel JD, Schilder RJ, Tahara M, Pfister SM, Witt O, Ladanyi M, Rudzinski ER, Nanda S, Childs BH, Laetsch TW, Hyman DM, Drilon A. Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1/2 clinical trials. Lancet Oncol. 2020 Apr;21(4):531-540. doi: 10.1016/S1470-2045(19)30856-3. Epub 2020 Feb 24.
PubMed 32105622Bielack SS, Cox MC, Nathrath M, Apel K, Blattmann C, Holl T, Jenewein R, Klenk U, Klothaki P, Muller-Abt P, Ortega-Lawerenz S, Reynolds M, Scheer M, Simon-Klingenstein K, Stegmaier S, Tupper R, Vokuhl C, von Kalle T. Rapid, complete and sustained tumour response to the TRK inhibitor larotrectinib in an infant with recurrent, chemotherapy-refractory infantile fibrosarcoma carrying the characteristic ETV6-NTRK3 gene fusion. Ann Oncol. 2019 Nov 1;30(Suppl_8):viii31-viii35. doi: 10.1093/annonc/mdz382.
PubMed 31738425
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study of the Oral TRK Inhibitor Larotrectinib in Pediatric Patients With Advanced Solid or Primary Central Nervous System Tumors
- ClinicalTrials.gov ID
- NCT02637687
- Acronym / sponsor ID
- SCOUT / 20290
- Conditions
- Solid Tumors Harboring NTRK Fusion
- Keywords
- Advanced solid tumors, Central nervous system (CNS) tumor, Extra-cranial tumor, Infantile fibrosarcoma (IFS), Neurotrophic tyrosine receptor kinase (NTRK), NTRK1, NTRK2, NTRK3
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 154 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 16 Dec 2015 (actual)
- Primary completion
- 20 Jul 2024 (actual)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 22 Dec 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is being done to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3). Larotrectinib blocks the actions of these NTRK genes in cancer cells and can therefore be used to treat cancer.
The first study part (Phase 1) is done to determine what dose level of larotrectinib is safe for children, how the drug is absorbed and changed by their bodies and how well the cancer responds to the drug. The main purpose of the second study part (Phase 2) is to investigate how well and how long different cancer types respond to the treatment with larotrectininb.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Phase 1 dose escalation | Experimental | Drug: Larotrectinib (Vitrakvi, BAY2757556)Patients will receive the different levels of dose on Day 1 (BID in accordance with the cohort assignment). Each cycle will consist of 28 days of continuous dosing. Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. (arm closed) |
| Phase 1 dose expansion | Experimental | Drug: Larotrectinib (Vitrakvi, BAY2757556)Patients who are enrolled in the expansion cohort, following the formal dose escalation phase of the study. Distinct from the Phase 1 dose escalation cohort, the Phase 1 expansion cohort will enroll pediatric patients with advanced solid or primary CNS tumors with a documented NTRK gene fusion, or in the case of IFS, CMN or SBC with documented ETV6 rearrangement by FISH or RT-PCR or a documented NTRK fusion by NGS. This expansion cohort will follow the same schedule of assessments as the dose escalation cohorts. (arm closed) |
| Phase 2: Patients with tumors bearing NTRK fusions (IFS)_Cohort 1 | Experimental | Drug: Larotrectinib (Vitrakvi, BAY2757556)Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing. Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. (arm closed) |
| Phase 2: Other extra-cranial solid tumors_Cohort 2 | Experimental | Drug: Larotrectinib (Vitrakvi, BAY2757556)Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing. Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. (arm closed) |
| Phase 2: Primary CNS tumors_Cohort 3 | Experimental | Drug: Larotrectinib (Vitrakvi, BAY2757556)Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing. Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. |
| Phase 2: Bone health assessment_sub-cohort | Experimental | Drug: Larotrectinib (Vitrakvi, BAY2757556)Patients will receive larotrectinib dose on Day 1 (BID in accordance with the cohort assignment) at the recommended Phase 2 dose as determined in the Phase 1 portion of this study. Each cycle will consist of 28 days of continuous dosing. Individual patients will continue daily larotrectinib dosing until PD, unacceptable toxicity, or other reason for treatment discontinuation. Patients in this group will undergo bone health assessments in addition to all other efficacy and safety assessments. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Phase 1 (Closed):
- Phase 2:
- -- Infants from birth and older at C1D1 with a locally advanced or metastatic infantile fibrosarcoma, patients with locally advanced infantile fibrosarcoma who would require, in the opinion of the investigator, disfiguring surgery or limb amputation to achieve a complete surgical resection; OR Birth through 21 years of age at C1D1 with a locally advanced or metastatic solid tumor or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies and for which no standard or available systemic curative therapy exists with a documented NTRK gene fusion (or in the case of infantile fibrosarcoma, congenital mesoblastic nephroma or secretory breast cancer with documented ETV6 rearrangement (or NTRK3 rearrangement after discussion with the sponsor) by FISH or RT-PCR. Patients with NTRK-fusion positive benign tumors are also eligible; OR Potential patients older than 21 years of age with a tumor diagnosis with histology typical of a pediatric patient and an NTRK fusion may be considered for enrollment following discussion between the local site Investigator and the Sponsor.
- Patients with primary CNS tumors or cerebral metastasis
- Karnofsky (those 16 years and older) or Lansky (those younger than 16 years) performance score of at least 50.
Key exclusion criteria (6 listed)
- Major surgery within 14 days (2 weeks) prior to C1D1
- Clinically significant active cardiovascular disease or history of myocardial infarction within 6 months prior to C1D1, ongoing cardiomyopathy; current prolonged QTc interval > 480 milliseconds
- Active uncontrolled systemic bacterial, viral, or fungal infection
- Current treatment with a strong CYP3A4 inhibitor or inducer. Enzyme-inducing anti-epileptic drugs (EIAEDs) and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed.
- Phase 2 only:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Larotrectinib (Vitrakvi, BAY2757556)Also: LOXO-101BAY2757556 will be administered orally as capsule or in liquid form over continuous 28-day cycles. | Drug | Phase 1 dose escalation, Phase 1 dose expansion, Phase 2: Bone health assessment_sub-cohort, Phase 2: Other extra-cranial solid tumors_Cohort 2, Phase 2: Patients with tumors bearing NTRK fusions (IFS)_Cohort 1, Phase 2: Primary CNS tumors_Cohort 3 |
Primary outcome
Phase 1: Number of participants in an assigned dose cohort with treatment emergent adverse events (TEAEs) by grade assessed by NCI-CTCAE v 4.03 who experience a DLT
Time frame: From Day 1 to Day 28 of Cycle 1 (1 Cycle=28 days)
DLT: Dose-limiting toxicity. NCI-CTCAE: National Cancer Institute-Common Terminology Criteria for Adverse Events.
Phase 1: Number of participants with TEAEs
Time frame: From first dose of larotrectinib up to 93 months
Phase 1: Severity of TEAEs
Time frame: From first dose of larotrectinib up to 93 months
Phase 2: Overall response rate (ORR) by IRRC
Time frame: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death, up to 76 months
Proportion of participants with a best overall response of complete response (CR) or partial response (PR) as determined by an independent radiology review committee (IRRC) based on Response Evaluation Criteria in Solid Tumours (RECIST) 1.1, Response Assessment in Neuro Oncology (RANO) or International Neuroblastoma Response Criteria (INRC) as appropriate to tumor type who express NTRK gene fusions.
22 secondary outcomes
Phase 1: Maximum concentration of larotrectinib in plasma (Cmax)
Time frame: Cohort 1 and 2: Cycle 1 Day 1 (C1D1) at 1 and 4 hours post-dose and C2D1 at pre-dose, and at 1 and 4 hours post-dose; Cohort 3 and Dose Expansion Cohort: C1D1 at 1 and 4 hours post-dose and C4D1 at pre-dose, 1 and 4 hours post-dose
Phase 1: Area under the concentration versus time curve from time 0 to t (AUC0-t) of larotrectinib in plasma
Time frame: Cohort 1 and 2: C1D1 at 1 and 4 hours post-dose and C2D1 at pre-dose, and at 1 and 4 hours post-dose; Cohort 3 and Dose Expansion Cohort: C1D1 at 1 and 4 hours post-dose and C4D1 at pre-dose, 1 and 4 hours post-dose
Phase 1: Oral clearance (CL/F)
Time frame: Cohort 1 and 2: C1D1 at 1 and 4 hours post-dose and C2D1 at pre-dose, and at 1 and 4 hours post-dose; Cohort 3 and Dose Expansion Cohort: C1D1 at 1 and 4 hours post-dose and C4D1 at pre-dose, 1 and 4 hours post-dos
Phase 1: Cerebral spinal fluid/plasma ratio of larotrectinib
Time frame: C1D1 in conjunction with the post-dose 1-hour PK sample
Phase 1: Maximum tolerated dose (MTD)
Time frame: From C1D1 to C1D28 of treatment of each participant in each of the assigned dose cohort, up to 16 months
Phase 1: Recommended dose for Phase 2
Time frame: From the date a participants from assigned Cohort was administered the first dose to the date of the last dose for the last patient from the dose escalation phase, up to 16 months
Phase 1: Overall Response Rate (ORR)
Time frame: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 93 months
Phase 1: Mean change from baseline in Pain scores as assessed by the Wong-Baker Faces scale
Time frame: Baseline and D1 of every cycle (1 Cycle=28 days), up to 93 months
Phase 1: Mean change in Health-related quality of life scores by PedsQL-Core
Time frame: Baseline and D1 of every cycle (1 Cycle=28 days), Up to 93 months
Phase 2: Best overall response (BOR)
Time frame: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 76 months
Phase 2: Duration of response (DOR)
Time frame: From start of first objective response of confirmed CR or PR to progression or death (due to any cause), up to 76 months
Phase 2: Proportion of patients with any tumor regression (i.e., any decrease from baseline of the longest diameters of target lesions) as a best response
Time frame: From first dose of Larotrectinib, up to 76 months
Phase 2: Progression-free survival (PFS)
Time frame: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 112 months
Phase 2: Overall survival (OS)
Time frame: From first dose of Larotrectinib to death (due to any cause), up to 112 months
Phase 2: Number of participants with Treatment emergent adverse events (TEAEs)
Time frame: From first dose of larotrectinib to discontinuation of treatment or death (due to any cayse), up to 112 months
Phase 2: Severity of adverse events as assessed by NCI-CTCAE grading V 4.03
Time frame: From first dose of larotrectinib to discontinuation of treatment or death (due to any cause), up to 112 months
Phase 2: Clinical Benefit Rate (CBR)
Time frame: From first dose of Larotrectinib to disease progression or subsequent therapy or surgical intervention or death (due to any cause), up to 76 months
Phase 2: Concordance coefficient
Time frame: From baseline/screening and if feasible end of treatment (EOT) and or PD and or at re-start of study treatment following a "drug holiday" and disease recurrence, up to 112 months
Phase 2: Post-operative tumor staging
Time frame: From first dose of Larotrectinib to surgical intervention, up to 112 months
Phase 2: Post-operative surgical margin assessment
Time frame: From first dose of Larotrectinib to surgical intervention, up to 112 months
Phase 2: Pre-treatment surgical plan to preserve function and cosmetic outcome
Time frame: From first dose of Larotrectinib to surgical intervention, up to 112 months
Phase 2: Post-treatment plans to conserve function and cosmetic outcome
Time frame: From surgical intervention to subsequent therapy, up to 112 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2025.
- Derived from this study: PubMed 42614029Mascarenhas L, Ollis C, Singh Y, Menachery L, Burcoveanu DI, De La Cuesta E, van Tilburg CM, Laetsch TW. Cancer control after pausing treatment with larotrectinib in responding children with sarcoma and a TRK fusion: a plain language summary. Future Oncol. 2026 Aug 19:1-10. doi: 10.1080/14796694.2026.2711519. Online ahead of print.
- Derived from this study: PubMed 40341199Ajani JA, D'Amico TA, Bentrem DJ, Corvera CU, Das P, Enzinger PC, Enzler T, Gerdes H, Gibson MK, Grierson P, Gupta G, Hofstetter WL, Ilson DH, Jalal S, Kim S, Kleinberg LR, Klempner S, Lacy J, Lee B, Licciardi F, Lloyd S, Ly QP, Matsukuma K, McNamara M, Merkow RP, Miller AM, Mukherjee S, Mulcahy MF, Perry KA, Pimiento JM, Reddi DM, Reznik S, Roses RE, Strong VE, Su S, Uboha N, Wainberg ZA, Willett CG, Woo Y, Yoon HH, McMillian NR, Stein M. Gastric Cancer, Version 2.2025, NCCN Clinical Practice Guidelines In Oncology. J Natl Compr Canc Netw. 2025 May;23(5):169-191. doi: 10.6004/jnccn.2025.0022.
- Derived from this study: PubMed 40267388Brose MS, Westphalen CB, Pan X, Bernard-Gauthier V, Kurtinecz M, Guo H, Aris V, Brett NR, Majdi A, Subbiah V, Pennell NA, Kehl KL, Drilon A. Larotrectinib Compared With Real-World Non-Tropomyosin Receptor Kinase Inhibitor Therapies in Patients With Tropomyosin Receptor Kinase Fusion Cancer. JCO Precis Oncol. 2025 Apr;9:e2400500. doi: 10.1200/PO-24-00500. Epub 2025 Apr 23.
- Derived from this study: PubMed 39869835Mascarenhas L, DuBois SG, Albert CM, Bielack S, Orbach D, Federman N, Geoerger B, Nagasubramanian R, Zhang Y, Chisholm J, Gallego Melcon S, Goto H, Morgenstern DA, Owens C, Pappo AS, Perreault S, Schulte JH, Shukla N, Zwaan CM, Neu N, Bernard-Gauthier V, De La Cuesta E, van Tilburg CM, Laetsch TW. Elective Discontinuation of Larotrectinib in Pediatric Patients With TRK Fusion Sarcomas and Related Mesenchymal Tumors. J Clin Oncol. 2025 Apr;43(10):1180-1187. doi: 10.1200/JCO.24.00848. Epub 2025 Jan 27.
- Derived from this study: PubMed 38096907Nahon-Esteve S, Orbach D, Le Loarer F, Hofman P, Lassalle S. Fibroblastic orbital tumour with NTRK1 fusion transcript: when TRK inhibitors rescue surgery. Can J Ophthalmol. 2024 Aug;59(4):e407-e409. doi: 10.1016/j.jcjo.2023.11.008. Epub 2023 Dec 11. No abstract available.
- Derived from this study: PubMed 37934000Subbiah V, Burris HA 3rd, Kurzrock R. Revolutionizing cancer drug development: Harnessing the potential of basket trials. Cancer. 2024 Jan;130(2):186-200. doi: 10.1002/cncr.35085. Epub 2023 Nov 7.
- Derived from this study: PubMed 37769113Kummar S, Shen L, Hong DS, McDermott R, Keedy VL, Casanova M, Demetri GD, Dowlati A, Melcon SG, Lassen UN, Leyvraz S, Liu T, Moreno V, Patel J, Patil T, Mallick AB, Sousa N, Tahara M, Ziegler DS, Norenberg R, Arvis P, Brega N, Drilon A, Tan DSW. Larotrectinib efficacy and safety in adult patients with tropomyosin receptor kinase fusion sarcomas. Cancer. 2023 Dec 1;129(23):3772-3782. doi: 10.1002/cncr.35036. Epub 2023 Sep 28.
- Derived from this study: PubMed 36689698Bokemeyer C, Paracha N, Lassen U, Italiano A, Sullivan SD, Marian M, Brega N, Garcia-Foncillas J. Survival Outcomes of Patients With Tropomyosin Receptor Kinase Fusion-Positive Cancer Receiving Larotrectinib Versus Standard of Care: A Matching-Adjusted Indirect Comparison Using Real-World Data. JCO Precis Oncol. 2023 Jan;7:e2200436. doi: 10.1200/PO.22.00436.
- Derived from this study: PubMed 36606522Rudzinski ER, Drilon A, Moore A, Spinosa S, Willi M, Laetsch TW. Testing methods to diagnose TRK fusion cancer - a plain language summary and patient perspective. Future Oncol. 2022 Dec;18(38):4141-4151. doi: 10.2217/fon-2022-0863. Epub 2023 Jan 6.
- Derived from this study: PubMed 34850167Doz F, van Tilburg CM, Geoerger B, Hojgaard M, Ora I, Boni V, Capra M, Chisholm J, Chung HC, DuBois SG, Gallego-Melcon S, Gerber NU, Goto H, Grilley-Olson JE, Hansford JR, Hong DS, Italiano A, Kang HJ, Nysom K, Thorwarth A, Stefanowicz J, Tahara M, Ziegler DS, Gavrilovic IT, Norenberg R, Dima L, De La Cuesta E, Laetsch TW, Drilon A, Perreault S. Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors. Neuro Oncol. 2022 Jun 1;24(6):997-1007. doi: 10.1093/neuonc/noab274.
- Derived from this study: PubMed 33467570Bebb DG, Banerji S, Blais N, Desmeules P, Gill S, Grin A, Feilotter H, Hansen AR, Hyrcza M, Krzyzanowska M, Melosky B, Noujaim J, Purgina B, Ruether D, Simmons CE, Soulieres D, Torlakovic EE, Tsao MS. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Adults. Curr Oncol. 2021 Jan 15;28(1):523-548. doi: 10.3390/curroncol28010053.
- Derived from this study: PubMed 33435412Perreault S, Chami R, Deyell RJ, El Demellawy D, Ellezam B, Jabado N, Morgenstern DA, Narendran A, Sorensen PHB, Wasserman JD, Yip S. Canadian Consensus for Biomarker Testing and Treatment of TRK Fusion Cancer in Pediatric Patients. Curr Oncol. 2021 Jan 9;28(1):346-366. doi: 10.3390/curroncol28010038.
- Individual participant dataSharing planned: No. Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
45 sites in 21 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Larotrectinib (Vitrakvi, BAY2757556) in people with Solid Tumors Harboring NTRK Fusion, run by Bayer at 45 sites in 21 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 22 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 45 sites in United States, Australia, Canada, China, Germany and 16 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 1: Number of participants in an assigned dose cohort with treatment emergent adverse events (TEAEs) by grade assessed by NCI-CTCAE v 4.03 who experience a DLT (time frame: From Day 1 to Day 28 of Cycle 1 (1 Cycle=28 days)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02637687, which also lists 18 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2025.